THE CURRENT STATE OF HUMAN PLURIPOTENT STEM CELL-BASED THERAPIES.

Human pluripotent stem cells, namely human embryonic stem cells (hESC) and induced pluripotent stem cells (iPSC) have a theoretical potential to differentiate into any type of human cells, they can be maintained in vitro for extended periods of time and can easily be genetically manipulated and expanded to clinically relevant cell numbers. As such, they can potentially serve as off-the-shelf sources of normal or genetically modified cells for tissue regenerative and other medical applications, including immune therapies. This presentation will provide an overview of the current state of pluripotent stem cells-based therapies, with the particular focus on pluripotent stem cell-derived immune cells. Our group and others have established that T cells with increased affinity for cancer epitopes can be derived from genetically modified pluripotent stem cells. Such T cells phenotypically and functionally fully resemble normal T cells and kill haplotype matched target cells in a dose dependent manner in vitro and in vivo. Practical considerations suggest that the pluripotent derived stem cell-derived T cells can therapeutically be superior to genetically modified T cells taken from patients' blood, as they can be expanded to significantly greater numbers sufficient for treatment of a large number of patients and properly analyzed for genotoxic effects of vector integration through DNA sequencing approaches. These types of cell therapies hold promise for the treatment of a broad array of conditions, including cancer, HIV infection, and immune insufficiency disorders. [ABSTRACT FROM AUTHOR]